Successful allogeneic bone marrow transplant for Niemann–Pick disease type C2 is likely to be associated with a severe ‘graft versus substrate’ effect - Bonney, Denise K.; O’Meara, Anne; Shabani, Abdu; Imrie, Jackie; Bigger, Brian W.; Jones, Simon; Wraith, James E.; Wynn, Robert F. (Journal of inherited metabolic disease, 2010)
Keywords: Biochemistry; Bone Marrow Transplantation - adverse effects; Brain - pathology; Carrier Proteins - genetics; Case Report; Child Development; Drug Administration Schedule; Glutamic Acid; Glycoproteins - deficiency; Glycoproteins - genetics; Graft vs Host Disease - diagnosis; Graft vs Host Disease - drug therapy; Graft vs Host Disease - immunology; Graft vs Host Disease - therapy; Homozygote; Human Genetics; Humans; Immune Reconstitution Inflammatory Syndrome - immunology; Immunosuppressive Agents - administration & dosage; Immunosuppressive Agents - therapeutic use; Infant; Internal Medicine; Macrophages - drug effects; Macrophages - immunology; Macrophages - transplantation; Magnetic Resonance Imaging; Male; Medicine; Medicine & Public Health; Metabolic Diseases; Motor Skills; Mutation; Niemann-Pick Disease, Type C - genetics; Niemann-Pick Disease, Type C - surgery; Pediatrics; Phenotype; Pneumonia - immunology; Respiratory Tract Infections - immunology; Respiratory Tract Infections - therapy; Severity of Illness Index; Transplantation Conditioning - methods; Transplantation, Homologous; Treatment Outcome; Neonatal Jaundice; Graft Versus Host Disease; Alemtuzumab; Acute Graft Versus Host Disease; Bone Marrow Transplantation